Exciting new therapy for haemophilia

Agreement has been reached for the provision of a new and innovative treatment for people with haemophilia, it was confirmed at the annual Irish Haemophilia Society members’ conference this weekend.

Haemophilia is an inherited bleeding disorder where one of the clotting factors is missing – or present in lower amounts- and standard treatment for the past 40 years has been based on intravenous injections of the missing clotting factor.
In order to prevent joint damage, injections were administered three times per week before in 2018 an improved form of the intravenous injection was introduced which could be infused twice per week.

The new treatment, Emicizumab (Hemlibra), licenced by the European Medicines Agency, is a bi-specific monoclonal antibody which mimics the effect of the clotting factor VIII injections for patients with Haemophilia A. It will offer more protection against bleeding and, importantly, rather than requiring vein access, it is injected subcutaneously (under the skin) once a week or once every two weeks.

“This new treatment is a very significant step forward”, said Brian O’Mahony, Chief Executive of the Irish Haemophilia Society.  “It will provide greater protection for people with haemophilia from the risk of bleeding and it will be much easier to administer without the need for regular intravenous injections” he stated.

“The burden of venous access and central venous access devices, with their potential for infective, mechanical and other complications, will be lifted from families of children with severe factor VIII deficiency using Hemlibra for prophylaxis”, stated Dr Beatrice Nolan, Consultant Haematologist and director of the National Paediatric Haemophilia Treatment Centre, Children\’s hospital, Ireland, Crumlin.

The product will be available to all people with the severe form of Haemophilia A which is the most common type of the condition.

It has been made available following an agreement with the Haemophilia Product Selection and Monitoring Advisory Board, which procures medications for haemophilia in Ireland. The board includes haemophilia specialists, the Irish Haemophilia Society and officials from the Department of Health.